Introduction: Haemophilia A is a rare, inherited bleeding disorder characterised by a tendency to bleed due to a lack of coagulation factor VIII. The severe form of haemophilia A is accompanied by spontaneous bleeding in the joints and muscles, and the most common complication is haemophilic arthropathy. These patients are treated prophylactically or as needed with factor VIII, and since 2019, also prophylactically with the monoclonal antibody emicizumab. In routine clinical practice, data on health-related quality of life in adult patients with severe haemophilia A and haemophilic arthropathy treated with emicizumab are still limited. Purpose: The study aimed to assess the quality of life of adult patients with severe haemophilia A and haemophilic arthropathy without inhibitors before the introduction of prophylactic treatment with emicizumab and after one year of treatment with emicizumab in routine clinical practice. Methods: We conducted a prospective study using quantitative methodology at the Clinical Department of Haematology of the Internal Clinic of the University Medical Centre Ljubljana from 2020 to 2022. Health-related quality of life before and after one year of emicizumab prophylaxis was assessed using the Slovenian adaptation of the internationally validated SF-36v2 questionnaire and a paired t-test or Wilcoxon rank-sum test. Results: At the beginning of the research, the surveyed patients assessed their quality of life as poor compared to the general population, especially in the field of physical functioning, as the average points for the dimensions of the physical component were below 50 (from 39.3 to 45.9 points), where 50 presents norm for the general population. After one year of prophylaxis with emicizumab, the surveyed patients showed better results in all dimensions, and statistically significant in the dimension of physical functioning (44.5 ± 10.2 after emicizumab vs. 39.3 ± 6.8 before emicizumab, p=0.018), physical component summary (46.6 ± 9.6 vs. 42.4 ± 7.1, p=0.017), and social functioning (50.9 ± 9.7 vs. 46.4 ± 9.4, p=0.014). Discussion and conclusion: The results of our study suggest improved physical and social functioning in adult patients with haemophilia A and haemophilic arthropathy after one year of emicizumab prophylaxis. To improve healthcare and effectively monitor the introduction of new medications in haemophilia, it would be reasonable to establish systematic monitoring of quality of life and mental health, including symptoms of depression, in all patients with haemophilia.
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